EA201491412A1 - Соединения для лечения спинальной мышечной атрофии - Google Patents

Соединения для лечения спинальной мышечной атрофии

Info

Publication number
EA201491412A1
EA201491412A1 EA201491412A EA201491412A EA201491412A1 EA 201491412 A1 EA201491412 A1 EA 201491412A1 EA 201491412 A EA201491412 A EA 201491412A EA 201491412 A EA201491412 A EA 201491412A EA 201491412 A1 EA201491412 A1 EA 201491412A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
connections
muscular atrophy
spinal muscular
spinal
Prior art date
Application number
EA201491412A
Other languages
English (en)
Other versions
EA028344B1 (ru
Inventor
Гуанмин Чэнь
Амал Дакка
Гари Митчелл Карп
Чуньши Ли
Яна Нарасимхан
Николай Нарышкин
Марла Л. Виталл
Эллен Уэлч
Синь Чжао
Original Assignee
ПиТиСи ТЕРАПЬЮТИКС, ИНК.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ПиТиСи ТЕРАПЬЮТИКС, ИНК. filed Critical ПиТиСи ТЕРАПЬЮТИКС, ИНК.
Publication of EA201491412A1 publication Critical patent/EA201491412A1/ru
Publication of EA028344B1 publication Critical patent/EA028344B1/ru

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D513/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
    • C07D513/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
    • C07D513/04—Ortho-condensed systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D311/00—Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings
    • C07D311/02—Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings ortho- or peri-condensed with carbocyclic rings or ring systems
    • C07D311/76—Benzo[c]pyrans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/02—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
    • C07D405/04—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/14—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D407/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00
    • C07D407/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00 containing two hetero rings
    • C07D407/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D409/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings
    • C07D409/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D409/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D497/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having oxygen and sulfur atoms as the only ring hetero atoms
    • C07D497/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having oxygen and sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D497/04—Ortho-condensed systems

Abstract

Изобретение относится к соединениям, их композициям и их применениям для лечения спинальной мышечной атрофии.
EA201491412A 2012-01-26 2013-01-25 Соединения для лечения спинальной мышечной атрофии EA028344B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261591102P 2012-01-26 2012-01-26
PCT/US2013/023067 WO2013112788A1 (en) 2012-01-26 2013-01-25 Compounds for treating spinal muscular atrophy

Publications (2)

Publication Number Publication Date
EA201491412A1 true EA201491412A1 (ru) 2014-12-30
EA028344B1 EA028344B1 (ru) 2017-11-30

Family

ID=48873933

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201491412A EA028344B1 (ru) 2012-01-26 2013-01-25 Соединения для лечения спинальной мышечной атрофии

Country Status (13)

Country Link
US (1) US9399649B2 (ru)
EP (1) EP2809322B9 (ru)
JP (2) JP6193888B2 (ru)
KR (1) KR102064624B1 (ru)
CN (1) CN104203239B (ru)
BR (1) BR112014018027B1 (ru)
CA (1) CA2862084C (ru)
EA (1) EA028344B1 (ru)
ES (1) ES2733644T3 (ru)
HK (1) HK1202069A1 (ru)
MX (1) MX357834B (ru)
PL (1) PL2809322T3 (ru)
WO (1) WO2013112788A1 (ru)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112014016287B1 (pt) 2011-12-30 2022-08-09 F. Hoffmann - La Roche Ag Composto e composição farmacêutica
ES2733644T3 (es) * 2012-01-26 2019-12-02 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
WO2013119916A2 (en) 2012-02-10 2013-08-15 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
KR102099997B1 (ko) 2012-03-01 2020-04-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
CA2868026C (en) 2012-03-23 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
BR112015015075A2 (pt) * 2012-12-24 2019-01-15 Univ Ramot agentes para tratar doenças genéticas resultantes de mutações sem sentido e métodos para identificar as mesmas.
EP3035935B1 (en) 2013-08-19 2020-03-11 F. Hoffmann-La Roche AG Compounds for use in the prophylaxis and treatment of cancer
EP3082820B1 (en) 2013-12-19 2022-07-20 PTC Therapeutics, Inc. Methods for modulating the amount of rna transcripts
WO2015095446A1 (en) * 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
US10882868B2 (en) 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
MX2018005890A (es) * 2015-11-12 2018-08-15 Hoffmann La Roche Composiciones para tratar atrofia muscular espinal.
CA3005434A1 (en) 2015-11-16 2017-05-26 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein
KR20220100719A (ko) 2015-12-10 2022-07-15 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
JP6872550B2 (ja) 2015-12-10 2021-05-19 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 架橋されたピペリジン誘導体
JP6738044B2 (ja) * 2016-07-22 2020-08-12 Jnc株式会社 ベンゾピラン骨格を有する液晶性化合物、液晶組成物、および液晶表示素子
CA3043755A1 (en) 2016-11-28 2018-05-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
WO2018226622A1 (en) 2017-06-05 2018-12-13 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
EA202090034A1 (ru) 2017-06-14 2020-04-16 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы модификации сплайсинга рнк
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
CA3067592A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
KR102636384B1 (ko) 2017-08-04 2024-02-14 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
EA202092001A1 (ru) 2018-03-27 2021-01-29 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения болезни гентингтона
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
US20220287748A1 (en) * 2018-06-27 2022-09-15 Reborna Biosciences, Inc. Prophylactic or therapeutic agent for spinal muscular atrophy
SG11202012674PA (en) 2018-06-27 2021-01-28 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for treating huntington's disease
KR20210135241A (ko) 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
KR20210135507A (ko) 2019-02-06 2021-11-15 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5854142B2 (ja) * 1975-04-16 1983-12-02 株式会社興人 イソカルボスチリル誘導体の製造方法
US5089633A (en) * 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US6180625B1 (en) * 1998-03-24 2001-01-30 Novo Nordisk A/S Heterocyclic compounds regulating clotting
WO2003005025A1 (en) * 2001-07-03 2003-01-16 Biovitrum Ab Methods for identifying compounds modulating the activity of ppar-gamma
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
US20040077529A1 (en) * 2002-06-10 2004-04-22 Per-Fredrik Lehmann Urotensin II receptor agents
BR0312232A (pt) * 2002-06-28 2005-05-10 Vertex Pharma Inibidores de caspases e seus usos
JP2004168707A (ja) * 2002-11-20 2004-06-17 Bayer Cropscience Ag イソチアゾリルベンゾオキサジン誘導体および病害防除剤
US9068234B2 (en) 2003-01-21 2015-06-30 Ptc Therapeutics, Inc. Methods and agents for screening for compounds capable of modulating gene expression
WO2004111014A1 (en) 2003-06-06 2004-12-23 Vertex Pharmaceuticals Incorporated Pyrimidine derivatives as modulators of atp-binding cassette transporters
JP2008528496A (ja) 2005-01-21 2008-07-31 ジヤンセン・フアーマシユーチカ・ナームローゼ・フエンノートシヤツプ エストロゲン受容体のモジュレーターとして有用な新規な複素環式ベンゾ［ｃ］クロメン誘導体
KR100781704B1 (ko) 2005-04-20 2007-12-03 에스케이케미칼주식회사 피리딘 유도체와 이의 제조방법, 및 이를 포함하는약제조성물
AR059339A1 (es) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene
WO2008013997A2 (en) 2006-07-27 2008-01-31 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
KR20080091949A (ko) 2007-04-10 2008-10-15 에스케이케미칼주식회사 락톤형 피리딘 화합물을 포함하는 허혈성 질환의 예방 및치료용 약학조성물
JP5276878B2 (ja) * 2007-09-27 2013-08-28 富士フイルム株式会社 ベンゾオキサジノン系化合物の製造方法
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
BR112014016287B1 (pt) 2011-12-30 2022-08-09 F. Hoffmann - La Roche Ag Composto e composição farmacêutica
ES2733644T3 (es) * 2012-01-26 2019-12-02 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
WO2013119916A2 (en) 2012-02-10 2013-08-15 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
KR102099997B1 (ko) 2012-03-01 2020-04-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
CA2868026C (en) 2012-03-23 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy

Also Published As

Publication number Publication date
JP2015511224A (ja) 2015-04-16
KR20140127271A (ko) 2014-11-03
JP2017171667A (ja) 2017-09-28
US9399649B2 (en) 2016-07-26
CN104203239A (zh) 2014-12-10
ES2733644T3 (es) 2019-12-02
HK1202069A1 (en) 2015-09-18
CA2862084C (en) 2021-05-11
KR102064624B1 (ko) 2020-01-09
US20150126515A1 (en) 2015-05-07
PL2809322T3 (pl) 2019-11-29
JP6193888B2 (ja) 2017-09-06
EP2809322B1 (en) 2019-05-15
EP2809322B9 (en) 2019-10-30
BR112014018027A2 (ru) 2017-06-20
EA028344B1 (ru) 2017-11-30
EP2809322A4 (en) 2015-12-23
CN104203239B (zh) 2017-09-08
CA2862084A1 (en) 2013-08-01
BR112014018027B1 (pt) 2020-10-27
MX357834B (es) 2018-07-26
EP2809322A1 (en) 2014-12-10
MX2014008875A (es) 2014-10-14
WO2013112788A1 (en) 2013-08-01
BR112014018027A8 (pt) 2017-07-11

Similar Documents

Publication Publication Date Title
EA201491412A1 (ru) Соединения для лечения спинальной мышечной атрофии
EA201491766A1 (ru) Соединения для лечения спинальной мышечной атрофии
EA201491505A1 (ru) Соединения для лечения спинальной мышечной атрофии
EA201491617A1 (ru) Соединения для лечения спинальной мышечной атрофии
EA201890754A1 (ru) Соединения и способы их применения
EA201492002A1 (ru) Противовирусные соединения
EA201790273A1 (ru) Флагеллиновые композиции и их применение
EA201691070A1 (ru) Новые трициклические соединения в качестве противораковых средств
EA201890333A1 (ru) Противовирусные соединения
EA201691272A1 (ru) Тетрагидропиридопиразины в качестве модуляторов gpr6
EA201992133A1 (ru) Производные пиперидино-пиримидина для лечения вирусных инфекций
IN2014DN09346A (ru)
EA201591728A1 (ru) Гетероциклические соединения и способы их применения
EA201270257A1 (ru) Производные n1-сульфонил-5-фторпиримидинона
EA201590997A1 (ru) Соединения и способы их применения
EA201500383A1 (ru) Производные 1,2,4-триазина для лечения вирусных инфекций
EA201492056A1 (ru) Пиримидиниловые ингибиторы тирозинкиназы
EA201590887A1 (ru) Композиция
EA201690371A1 (ru) Пиридазиноновые соединения и их применение
EA201490423A1 (ru) СПОСОБЫ И КОМПОЗИЦИИ, ОТНОСЯЩИЕСЯ К p62, ДЛЯ ЛЕЧЕНИЯ И ПРОФИЛАКТИКИ РАКА
EA201290642A1 (ru) Соединения и способы
GEP201706710B (en) Imidazopyridazine derivatives as gabaa receptor modulators
EA201690783A1 (ru) Вещества, фармацевтическая композиция и методы применения при лечении воспалительных заболеваний
EA201270253A1 (ru) Производные n1-ацил-5-фторпиримидинона
CY1119470T1 (el) Γαλακταγωγες συνθεσεις με βαση φωσφατιδυλοσερινη

Legal Events

Date Code Title Description
MM4A Lapse of a eurasian patent due to non-payment of renewal fees within the time limit in the following designated state(s)

Designated state(s): AM AZ BY KZ KG TJ TM